Panel advising US regulator rejects MDMA as treatment for post-traumatic stress disorder

A panel of experts convened by the US Food and Drug Administration has resoundingly voted against the use of MDMA, better known as party drug ecstasy, as a treatment for post-traumatic stress disorder.

The ruling on Tuesday was a big setback for Lykos Therapeutics, the biotech developing the treatment, and the nascent psychedelics sector, which has received hundreds of millions of dollars of investment in recent years.

Following a nine-hour evidence session, the FDA advisory committee, composed of mental health and medical experts, voted by a margin of 9 to 2 that the available data did not prove that the drug was an effective treatment of PTSD in combination with therapy. In a second vote, the panel also concluded the benefits of the treatment did not outweigh the risks, with the vote carried by 10 to 1.

The FDA is due to make its final decision by mid-August. The panel’s vote is non-binding but typically holds significant sway with the agency and therefore will dramatically reduce the likelihood of the treatment getting approved, dashing the hopes of psychedelic advocates and investors.

Amy Emerson, chief executive of Lykos, said in a statement: “We are disappointed in today’s vote given the urgent unmet need in PTSD and appreciate that the committee faced a challenging and atypical assignment, which was to evaluate a therapeutic approach that combines drug therapy (MDMA) and psychological intervention.”

If approved, Lykos’s MDMA-based therapy treatment would have become the first new PTSD treatment in more than two decades. It also would have marked the first time a Schedule 1 drug, which the government deems as having no medical application and high potential for abuse, would have been approved as a regulated mental health treatment.

Earlier this year, Lykos — which was spun out of the Multidisciplinary Association of Psychedelic Studies, a non-profit — raised $100mn from a group of 10 investors, including the charitable foundation run by hedge fund billionaire Steven Cohen and his wife, Alexandra.

In the second of two phase 3 trials, the Lykos treatment showed 71 per cent of people who had received its MDMA-based therapy improved sufficiently to no longer meet the criteria for PTSD diagnosis, compared with 48 per cent in the placebo group. Patients received the drug over three eight-hour therapy sessions.

But the FDA panel was unconvinced, voting against endorsing the effectiveness of the treatment, citing issues with the trial design and data collection.

Rajesh Narendran, professor in radiology and psychiatry at the University of Pittsburgh School of Medicine who chaired the panel, said issues with the trial design made the data “meaningless”.

“There [were] just too many problems with [the study],” added Satish Iyengar, a statistics professor at the University of Pittsburgh.

Tiffany Farchione, an FDA official, earlier told the panel that Lykos had failed to collect data on side-effects of the drug associated with abuse, which the company had deemed positive, such as “euphoria” or “elated mood”, meaning it would be difficult to accurately label the drug. Farchione said the data collection issues “may have the greatest impact” on the agency’s decision.

David Millis, a clinical reviewer for the FDA, also raised concerns about “double-blinding” trial participants because of the powerful hallucinatory effects of MDMA. “Although we do have two positive studies, the results are in the context” of participants knowing they were on the drug, Millis said. Farchione also noted the trial had not adequately assessed the effect of psychotherapy on the treatment as well as the potential cardiology risks from the treatment.

Alia Lilienstein, Lykos’s senior medical director, pushed back against the criticism during the evidence session. Prior to the hearing, two influential non-profits — the Institute for Clinical and Economic Review and an offshoot of the American Psychological Association — had also criticised the Lykos treatment.